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Questions and Answers
What is the main approach used in gene therapy for diseases caused by recessive mutations?
What is the main approach used in gene therapy for diseases caused by recessive mutations?
- Complementing the defective gene (correct)
- Introducing a normal gene
- Targeting the protein product
- Silencing the normal gene
What strategy is typically employed for autosomal dominant genetic diseases in gene therapy?
What strategy is typically employed for autosomal dominant genetic diseases in gene therapy?
- Introducing a normal gene
- Activating the mutant protein
- Silencing the defective gene (correct)
- Increasing expression of the pathogenic gene
Which type of therapeutic RNA inhibits gene function by controlling gene function?
Which type of therapeutic RNA inhibits gene function by controlling gene function?
- mRNAs
- gRNAs
- siRNAs (correct)
- RNA aptamers
In gene therapy, what are RNAs that reprogram genetic information and include trans-splicing ribozymes used for?
In gene therapy, what are RNAs that reprogram genetic information and include trans-splicing ribozymes used for?
What is the main characteristic of siRNAs used in RNA interference?
What is the main characteristic of siRNAs used in RNA interference?
Which type of therapeutic RNA is used for DNA modification in gene therapy?
Which type of therapeutic RNA is used for DNA modification in gene therapy?
What is the main function of Aptamers in cancer therapy?
What is the main function of Aptamers in cancer therapy?
How are SiRNAs generated by the ribonuclease?
How are SiRNAs generated by the ribonuclease?
What is the main purpose of Ribozymes in gene therapy?
What is the main purpose of Ribozymes in gene therapy?
Which ribozyme motif is characterized by its ability for self-cleavage of a particular phosphodiester bond?
Which ribozyme motif is characterized by its ability for self-cleavage of a particular phosphodiester bond?
What is the special characteristic of Group I and Group II intron ribozymes?
What is the special characteristic of Group I and Group II intron ribozymes?
Which disease has Ribozymes been clinically trialed for according to the text?
Which disease has Ribozymes been clinically trialed for according to the text?
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